Abstract
While mortality is low for intraocular retinoblastoma patients in the developed world who receive aggressive multimodal therapy, partial or full loss of vision occurs in approximately 50% of patients with advanced bilateral retinoblastoma. Therapies that preserve vision and reduce late effects are needed. Because clinical trials for retinoblastoma are difficult due to the young age of the patient population and relative rarity of the disease, robust preclinical testing of new therapies is critical. The last decade has seen advances towards identifying new therapies including the development of animal models of retinoblastoma for preclinical testing, progress in local drug delivery to reach intraocular targets, and improved understanding of the underlying biological mechanisms that give rise to retinoblastoma. This review discusses advances in these areas, with a focus on discovery and development of small molecules for the treatment of retinoblastoma, including novel targeted therapeutics such as inhibitors of the MDMX-p53 interaction (nutlin-3a), histone deacetylase (HDAC) inhibitors, and spleen tyrosine kinase (SYK) inhibitors.
Keywords: Chemoreduction, Ocular drug delivery, Retinoblastoma.
Mini-Reviews in Medicinal Chemistry
Title:Progress in Small Molecule Therapeutics for the Treatment of Retinoblastoma
Volume: 16 Issue: 6
Author(s): Eleanor M. Pritchard, Michael A. Dyer and R. Kiplin Guy
Affiliation:
Keywords: Chemoreduction, Ocular drug delivery, Retinoblastoma.
Abstract: While mortality is low for intraocular retinoblastoma patients in the developed world who receive aggressive multimodal therapy, partial or full loss of vision occurs in approximately 50% of patients with advanced bilateral retinoblastoma. Therapies that preserve vision and reduce late effects are needed. Because clinical trials for retinoblastoma are difficult due to the young age of the patient population and relative rarity of the disease, robust preclinical testing of new therapies is critical. The last decade has seen advances towards identifying new therapies including the development of animal models of retinoblastoma for preclinical testing, progress in local drug delivery to reach intraocular targets, and improved understanding of the underlying biological mechanisms that give rise to retinoblastoma. This review discusses advances in these areas, with a focus on discovery and development of small molecules for the treatment of retinoblastoma, including novel targeted therapeutics such as inhibitors of the MDMX-p53 interaction (nutlin-3a), histone deacetylase (HDAC) inhibitors, and spleen tyrosine kinase (SYK) inhibitors.
Export Options
About this article
Cite this article as:
Pritchard M. Eleanor, Dyer A. Michael and Guy Kiplin R., Progress in Small Molecule Therapeutics for the Treatment of Retinoblastoma, Mini-Reviews in Medicinal Chemistry 2016; 16 (6) . https://dx.doi.org/10.2174/1389557515666150722100610
DOI https://dx.doi.org/10.2174/1389557515666150722100610 |
Print ISSN 1389-5575 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5607 |
Call for Papers in Thematic Issues
Bioprospecting of Natural Products as Sources of New Multitarget Therapies
According to the Convention on Biological Diversity, bioprospecting is the exploration of biodiversity and indigenous knowledge to develop commercially valuable products for pharmaceutical and other applications. Bioprospecting involves searching for useful organic compounds in plants, fungi, marine organisms, and microorganisms. Natural products traditionally constituted the primary source of more than ...read more
Computational Frontiers in Medicinal Chemistry
The thematic issue "Computational Frontiers in Medicinal Chemistry" provides a robust platform for delving into state-of-the-art computational methodologies and technologies that significantly propel advancements in medicinal chemistry. This edition seeks to amalgamate top-tier reviews spotlighting the latest trends and breakthroughs in the fusion of computational approaches, including artificial intelligence (AI) ...read more
Natural Products and Dietary Supplements in Alleviation of Metabolic, Cardiovascular, and Neurological Disorders
Metabolic disorders like diabetes, obesity, inflammation, oxidative stress, cancer etc, cardiovascular disorders like angina, myocardial infarction, congestive heart failure etc as well as neurological disorders like Alzheimer?s, Parkinson?s, Epilepsy, Depression, etc are the global burden. They covered the major segment of the diseases and disorders from which the human community ...read more
Natural Products in Drug Discovery
Natural products have always been one of the important ways of drug discovery due to their novel skeleton and diverse functional group characteristics. According to statistics, between 1981 and 2019, the FDA approved a total of 1,394 small molecule drugs for marketing, of which 930 marketed drugs originated from the ...read more
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
Related Articles
-
Mesenchymal Stromal Cell Therapy for Cardio Renal Disorders
Current Pharmaceutical Design Colon Cancer Therapy: Recent Developments in Nanomedicine to Improve the Efficacy of Conventional Chemotherapeutic Drugs
Anti-Cancer Agents in Medicinal Chemistry Targeting Nanomedicine to Brain Tumors: Latest Progress and Achievements
Current Pharmaceutical Design Possible Role of DNA Methylation in the Induction of Systemic Lupus Erythematosus
Current Rheumatology Reviews Phosphoprotein Phosphatase 1 Complexes in Spermatogenesis
Current Molecular Pharmacology Telomerase Modulation in Therapeutic Approach
Current Pharmaceutical Design A Closer Look at Protein Transduction Domains as a Tool in Drug Delivery
Current Nanoscience Cyclin Dependent Kinase 1 Inhibitors: A Review of Recent Progress
Current Medicinal Chemistry Gestational Trophoblastic Neoplasia, an Ancient Disease: New Light and Potential Therapeutic Targets
Anti-Cancer Agents in Medicinal Chemistry Editorial [Hot Topic: The RB-Pathway in Cancer (Executive Editor: Erik Knudsen)]
Current Molecular Medicine Thiazolidinediones Anti-Inflammatory and Anti-Atherosclerotic Effects in Type 2 Diabetes Mellitus
Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry E2F1-Mediated Apoptosis as a Target of Cancer Therapy
Current Molecular Pharmacology Translocator Protein (TSPO) Role in Aging and Alzheimer’s Disease
Current Aging Science Molecular Approaches Target to Immunotherapy for HPV-Associated Cancers
Current Cancer Drug Targets Clinical Approaches Toward Tumor Angiogenesis: Past, Present and Future
Current Pharmaceutical Design Targeting Phospholipase D-mediated Survival Signals in Cancer
Current Signal Transduction Therapy Potential Utilization of Bystander / Abscopal-Mediated Signal Transduction Events in the Treatment of Solid Tumors
Current Signal Transduction Therapy Imaging Endogenous Neurotransmitters In Vivo with Positron Emission Tomography Displacement Studies
Current Psychopharmacology Omega-3 Polyunsaturated Fatty Acids and Cancer
Anti-Cancer Agents in Medicinal Chemistry Tumor-Targeting Peptides: Ligands for Molecular Imaging and Therapy
Anti-Cancer Agents in Medicinal Chemistry